Ten children with seven differing subtypes of Leigh syndrome,
ranging in age from 1-13 years, were treated with EPI-743. All ten
children exhibited reversal of disease progression as measured by
four different disease-relevant metrics. The clinical response was
durable over 180 days. No significant safety events were
observed.

The results of the clinical trial were published on-line
September 10, 2012 in the journal Molecular Genetics and
Metabolism.

Findings obtained in this prospective phase 2 controlled study
confirm previous clinical results obtained in the United States and
Europe.

Leigh syndrome is an inherited lethal, progressive,
predominately pediatric, neuromuscular disorder for which there are
no approved treatments. Initially described in 1951, the hallmarks
of the disease include bilateral necrosis (death) of central
nervous system regions responsible for the control of breathing and
other neurologic functions. Leigh syndrome belongs to a large
family of disorders identified as "mitochondrial disease." The
disorders share as a common biochemical feature defects in cellular
energy metabolism.

EPI-743 is an orally bioavailable small molecule being developed
by Edison Pharmaceuticals for the treatment of Leigh syndrome and
other inherited mitochondrial diseases. EPI-743 is a member of the
para-benzoquinone class of drugs. It serves as a cofactor for the
novel drug target– NADPH quinone oxidase 1 (NQO1). Through a
redox-based mechanism, EPI-743 augments endogenous glutathione
biosynthesis– essential for the control of oxidative
stress.

Results obtained in this trial represent the first published
report of disease reversal in an otherwise progressive and fatal
neurological disease.

Edison Pharmaceuticals

Edison Pharmaceuticals is a specialty pharmaceutical company
dedicated to developing treatments for children with orphan
mitochondrial diseases.

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